scholarly journals Cost-Effectiveness of Short-Course Radiation Plus Temozolomide for the Treatment of Newly Diagnosed Glioblastoma Among Elderly Patients in China and the United States

2021 ◽  
Vol 12 ◽  
Author(s):  
Jigang Chen ◽  
Xin Tong ◽  
Mingyang Han ◽  
Songfeng Zhao ◽  
Linjin Ji ◽  
...  
Keyword(s):  
United States ◽  
Cost Effectiveness ◽  
Elderly People ◽  
Cost Effective ◽  
The United States ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Newly Diagnosed ◽  
Life Years ◽  
The Us

Background: Glioblastoma multiforme (GBM) is a fatal type of brain tumor with a high incidence among elderly people. Temozolomide (TMZ) has proven to be an effective chemotherapeutic agent with significant survival benefits. This study aimed to evaluate the economic outcomes of radiotherapy (RT) and TMZ for the treatment of newly diagnosed GBM in elderly people in the United States (US) and China.Methods: A partitioned survival model was constructed for RT plus TMZ and RT alone among patients with methylated and unmethylated tumor status. Base case calculations and one-way and probabilistic sensitivity analyses were performed. Life-years, quality-adjusted life-years (QALYs), costs (in 2021 US dollars [$] and Chinese Yuan Renminbi [¥]), and incremental cost-effectiveness ratios (ICERs) were calculated.Results: RT plus TMZ was found to be associated with significantly higher costs and QALYs in all groups. Only US patients with methylated status receiving RT plus TMZ had an ICER ($89358.51) less than the willingness-to-pay (WTP) threshold of $100000 per QALY gained when compared with receiving RT alone. When the WTP threshold ranged from $100000 to $150000 from the US perspective, the probability of RT plus TMZ being cost-effective increased from 80.5 to 99.8%. The cost of TMZ must be lower than ¥120 per 20 mg for RT plus TMZ to be cost-effective among patients with methylated tumor status in China.Conclusion: RT plus TMZ was not cost-effective in China, and a reduction in the TMZ price was justified. However, it is highly likely to be cost-effective for patients with methylated tumor status in the US.

scholarly journals Cost-effectiveness and health impact of lung cancer screening with low-dose computed tomography for never smokers in Japan and the United States: a modelling study

2022 ◽  
Vol 22 (1) ◽  
Author(s):  
Akiko Kowada
Keyword(s):  
United States ◽  
Lung Cancer ◽  
Cost Effectiveness ◽  
Lung Cancer Screening ◽  
Cost Effective ◽  
The United States ◽  
Sensitivity Analyses ◽  
Life Years ◽  
Never Smokers ◽  
Low Dose Computed Tomography

Abstract Background Never smokers in Asia have a higher incidence of lung cancer than in Europe and North America. We aimed to assess the cost-effectiveness of lung cancer screening with low-dose computed tomography (LDCT) for never smokers in Japan and the United States. Methods We developed a state-transition model for three strategies: LDCT, chest X-ray (CXR), and no screening, using a healthcare payer perspective over a lifetime horizon. Sensitivity analyses were also performed. Main outcomes were costs, quality-adjusted life-years (QALYs), life expectancy life-years (LYs), incremental cost-effectiveness ratios (ICERs), and deaths from lung cancer. The willingness-to-pay level was US$100,000 per QALY gained. Results LDCT yielded the greatest benefits with the lowest cost in Japan, but the ICERs of LDCT compared with CXR were US$3,001,304 per QALY gained for American men and US$2,097,969 per QALY gained for American women. Cost-effectiveness was sensitive to the incidence of lung cancer. Probabilistic sensitivity analyses demonstrated that LDCT was cost-effective 99.3–99.7% for Japanese, no screening was cost-effective 77.7% for American men, and CXR was cost-effective 93.2% for American women. Compared with CXR, LDCT has the cumulative lifetime potential for 60-year-old Japanese to save US$117 billion, increase 2,339,349 QALYs and 3,020,102 LYs, and reduce 224,749 deaths, and the potential for 60-year-old Americans to cost US$120 billion, increase 48,651 QALYs and 67,988 LYs, and reduce 2,309 deaths. Conclusions This modelling study suggests that LDCT screening for never smokers has the greatest benefits and cost savings in Japan, but is not cost-effective in the United States. Assessing the risk of lung cancer in never smokers is important for introducing population-based LDCT screening.

Financial implications of dialysis modalities in the developing world: A Chinese perspective

2020 ◽  
Vol 40 (2) ◽  
pp. 193-201
Author(s):  
Jing Liu ◽  
David W Hutton ◽  
Yonghong Gu ◽  
Yao Hu ◽  
Yi Li ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Direct Cost ◽  
Cost Effective ◽  
The United States ◽  
Direct Costs ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Published Data ◽  
Life Years ◽  
The Cost

Background: End-stage renal disease has been imposing a heavy economic burden on public health; however, few studies have been performed on the cost-effectiveness of dialysis modalities. We aim to estimate the cost-effectiveness of different dialysis modalities in China. Methods: Cost-effectiveness analyses were performed using Markov models based on published data of hemodialysis (HD) and peritoneal dialysis (PD) modalities in China. Sensitivity analyses were conducted to identify key variables influencing the results. Results: Over a 10-year time horizon, the base-case cost-effectiveness analysis indicated that PD-first absolutely dominated the HD-first option by gaining 0.13 more quality-adjusted life years (QALYs) and costing RMB ¥81,081 less. When using reported mortality of HD and PD from the United States, the PD-first option still dominated HD-first with higher QALYs and lower costs. Sensitivity analyses revealed that the results were more sensitive to the direct cost of HD, utility of HD, utility of PD, direct cost of PD, PD mortality, and HD mortality, while less sensitive to the indirect costs and transition probabilities. The HD utility needed to be at least 0.148 higher than PD utility for HD to be cost-effective. PD was about 72% likely to be considered cost-effective compared with HD, regardless of the willingness-to-pay for QALYs. Conclusion: PD appears to be more cost-effective than HD in China, and the major influential factors on the cost-effectiveness are the direct costs of HD, utility of HD, utility of PD, direct costs of PD, PD mortality, and HD mortality.

Cost-effectiveness of mobile health-based integrated care for atrial fibrillation: Model development and data analysis (Preprint)

2021 ◽  
Author(s):  
Xueyan Luo ◽  
Wei Xu ◽  
Quan Yuan ◽  
Han Lai ◽  
Chunji Huang
Keyword(s):  
Atrial Fibrillation ◽  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Integrated Care ◽  
Mobile Health ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Life Years ◽  
Mhealth Technology

BACKGROUND Mobile health (mhealth) technology is increasingly used in disease management. Using mhealth tools to integrate and streamline care was found to improve atrial fibrillation (AF) patients’ clinical outcomes. OBJECTIVE This study aimed to investigate the potential clinical and health economic outcomes of mhealth-based integrated care for AF from the perspective of a public healthcare provider in China. METHODS A Markov model was designed to compare outcomes of mhealth-based care and usual care in a hypothetical cohort of AF patients in China. The time horizon was 30 years with monthly cycles. Model outcomes measured were direct medical cost, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs). Sensitivity analyses were conducted to examine the robustness of base-case results. RESULTS In the base-case analysis, mhealth-based care gained higher QALYs of 0.0818 with an incurred cost of USD1,778. Using USD33,438 per QALY (three times gross domestic product) as the willingness-to-pay threshold, mhealth-based care was cost-effective, with an ICER of USD21,739 per QALY. The one-way sensitivity analysis found compliance to mhealth-based care had the greatest impact on the ICER. In probabilistic sensitivity analysis, mhealth-based care was accepted as cost-effective in 80.91% of 10,000 iterations. CONCLUSIONS This study suggested that the use of mhealth technology in streamlining and integrating care for AF patients was cost-effective in China.

scholarly journals Milk Powder Fortified with Potassium and Phytosterols to Decrease the Risk of Cardiovascular Events among the Adult Population in Malaysia: A Cost-Effectiveness Analysis

Nutrients ◽  
2019 ◽  
Vol 11 (6) ◽  
pp. 1235
Author(s):  
Anita E. Gandola ◽  
Livia Dainelli ◽  
Diane Zimmermann ◽  
Maznah Dahlui ◽  
Patrick Detzel
Keyword(s):  
Cost Effectiveness ◽  
Adult Population ◽  
Grey Literature ◽  
Milk Powder ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Discounted Cost ◽  
Life Years ◽  
The Impact

This study evaluated the cost-effectiveness of the consumption of a milk powder product fortified with potassium (+1050.28 mg/day) and phytosterols (+1200 mg/day) to lower systolic blood pressure and low-density lipoprotein cholesterol, respectively, and, therefore, the risk of myocardial infarction (MI) and stroke among the 35–75-year-old population in Malaysia. A Markov model was created against a do-nothing option, from a governmental perspective, and with a time horizon of 40 years. Different data sources, encompassing clinical studies, practice guidelines, grey literature, and statistical yearbooks, were used. Sensitivity analyses were performed to evaluate the impact of uncertainty on the base case estimates. With an incremental cost-effectiveness ratio equal to international dollars (int$) 22,518.03 per quality-adjusted life-years gained, the intervention can be classified as very cost-effective. If adopted nationwide, it would help prevent at least 13,400 MIs, 30,500 strokes, and more than 10,600 and 17,100 MI- and stroke-related deaths. The discounted cost savings generated for the health care system by those who consume the fortified milk powder would amount to int$8.1 per person, corresponding to 0.7% of the total yearly health expenditure per capita. Sensitivity analyses confirmed the robustness of the results. Together with other preventive interventions, the consumption of milk powder fortified with potassium and phytosterols represents a cost-effective strategy to attenuate the rapid increase in cardiovascular burden in Malaysia.

Cost-effectiveness of digital cognitive behavioral therapy (Sleepio) for insomnia: a Markov simulation model in the United States

SLEEP ◽  
2020 ◽  
Author(s):  
Michael Darden ◽  
Colin A Espie ◽  
Jenna R Carl ◽  
Alasdair L Henry ◽  
Jennifer C Kanady ◽  
...  
Keyword(s):  
United States ◽  
Cost Effectiveness ◽  
Cognitive Behavioral Therapy ◽  
Behavioral Therapy ◽  
Cost Effective ◽  
The United States ◽  
Cognitive Behavioral ◽  
Life Years ◽  
Insomnia Treatment ◽  
Group Cbt

Abstract Study Objectives To examine the cost-effectiveness and potential net monetary benefit (NMB) of a fully automated digital cognitive behavioral therapy (CBT) intervention for insomnia compared with no insomnia treatment in the United States (US). Similar relative comparisons were made for pharmacotherapy and clinician-delivered CBT (individual and group). Methods We simulated a Markov model of 100,000 individuals using parameters calibrated from the literature including direct (treatment) and indirect costs (e.g. insomnia-related healthcare expenditure and lost workplace productivity). Health utility estimates were converted into quality-adjusted life years (QALYs) and one QALY was worth $50,000. Simulated individuals were randomized equally to one of five arms (digital CBT, pharmacotherapy, individual CBT, group CBT, or no insomnia treatment). Sensitivity was assessed by bootstrapping the calibrated parameters. Cost estimates were expressed in 2019 US dollars. Results Digital CBT was cost beneficial when compared with no insomnia treatment and had a positive NMB of $681.06 (per individual over 6 months). Bootstrap sensitivity analysis demonstrated that the NMB was positive in 94.7% of simulations. Relative to other insomnia treatments, digital CBT was the most cost-effective treatment because it generated the smallest incremental cost-effectiveness ratio (−$3,124.73). Conclusions Digital CBT was the most cost-effective insomnia treatment followed by group CBT, pharmacotherapy, and individual CBT. It is financially prudent and beneficial from a societal perspective to utilize automated digital CBT to treat insomnia at a population scale.

scholarly journals P14.12 The cost-effectiveness of tumor-treating fields in patients with newly diagnosed glioblastoma

2019 ◽  
Vol 21 (Supplement_3) ◽  
pp. iii68-iii69
Author(s):  
X Armoiry ◽  
P Auguste ◽  
C Dussart ◽  
J Guyotat ◽  
M Connock
Keyword(s):  
Cost Effectiveness ◽  
Cost Effective ◽  
Survival Model ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Tumor Treating Fields ◽  
Kaplan Meier ◽  
Life Years ◽  
Secondary Analyses ◽  
The Cost

Abstract BACKGROUND The addition of novel therapy “Tumor-Treating fields” (TTF) to standard radio-chemotherapy with Temozolomide (TMZ) has recently shown superiority over conventional TMZ regimen in patients with glioblastoma. Despite the clinical benefit of TTF, there is a strong concern regarding the cost of this new treatment. A first cost-effectiveness analysis, which was published in 2016, was based on effectiveness outcomes from an interim analysis of the pivotal trial and used a “standard” Markov model. Here, we aimed to update the cost-effectiveness evaluation using a partitioned survival model design and using the latest effectiveness data. MATERIAL AND METHODS A partitioned survival model was developed with three mutually exclusive health states: stable disease, progressive disease, and dead. Parametric models were fitted to the Kaplan-Meier data for overall and progression-free survival. These generated clinically plausible extrapolations beyond the observed data. The perspective of the French national health insurance was adopted and the time horizon was 20 years. Base case results were expressed as cost/life-years (LY) gained (LYG). Secondary analyses were undertaken, with the results presented as cost/per quality adjusted life years (QALY) gained. Last, we undertook deterministic and probabilistic sensitivity analyses. RESULTS After applying 4% annual discounting of benefits and costs, the base case model generated incremental benefit of 0.507 LY at a incremental cost of €258,695 yielding an incremental cost effectiveness ratio (ICER) of €510,273 / LYG. Secondary analyses yielded an ICER of €667,173/QALY. Sensitivity analyses and bootstrapping methods showed the model was relatively robust. The model was sensitive to TTF device costs and the parametric model fitted to the Kaplan-Meier data for overall survival. The cost-effectiveness acceptability curve showed TTF has 0% of being cost-effective under conventional thresholds. CONCLUSION Using a partitioned survival model, uprated costs and more mature survival outcomes, TTF when compared to standard radio-chemotherapy with TMZ is not likely to be cost-effective. This has major implications in terms of access of newly eligible patients

Cost-effectiveness of once weekly carfilzomib 70 mg/m2 plus dexamethasone in patients with relapsed and refractory multiple myeloma in the United States.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e18356-e18356
Author(s):  
Shaji Kumar ◽  
Istvan Majer ◽  
Sumeet Panjabi ◽  
Jean Malacan ◽  
Rohan Medhekar ◽  
...  
Keyword(s):  
Health Care ◽  
Multiple Myeloma ◽  
Cost Effectiveness ◽  
Progression Free Survival ◽  
Cost Effective ◽  
The United States ◽  
Sensitivity Analyses ◽  
Refractory Multiple Myeloma ◽  
Life Years ◽  
Lifetime Costs

e18356 Background: Carfilzomib plus dexamethasone (Kd) dosed once weekly at 70 mg/m2 (QW Kd70) was recently approved in the US for treating patients with relapsed and refractory multiple myeloma (RRMM). To assess the cost-effectiveness (CE) of QW Kd70 vs twice weekly Kd dosed at 27 mg/m2 (BIW Kd27), data from the phase 3 ARROW trial, which directly compared these regimens in patients with 2-3 prior lines of therapy were used. Methods: A partitioned survival model was developed for the CE analysis. Time to treatment discontinuation, progression-free survival, and overall survival (OS) were estimated from the ARROW trial. Long-term OS was extrapolated using Surveillance Epidemiology and End Results registry data after matching characteristics of patients in the registry and ARROW trial. Direct costs were estimated from a US health care payer perspective. Utilities collected in the ARROW trial using the five-level version of the EuroQol questionnaire (EQ-5D-5L) were applied to estimate the quality-adjusted life years (QALYs). Uncertainty was explored using sensitivity analyses. Two subgroups of patients refractory to lenalidomide or bortezomib were assessed. Main outcomes were mean life-years (LYs), QALYs, lifetime costs, and incremental cost-effectiveness ratios (ICERs). Results: For QW Kd70 and BIW Kd27, the model predicted mean LYs of 4.17 and 3.07 years, QALYs of 2.98 and 2.03 years, and mean total lifetime costs of $444,563 and $373,364, respectively. The incremental LYs gain, QALY gain, and incremental costs of QW Kd70 vs BIW Kd27 were estimated to be 1.10 years, 0.95 year, and $71,199, respectively, resulting in an ICER of $64,595 per LY gained and $75,204 per QALY gained. For patients refractory to lenalidomide and bortezomib, similar results were found with ICERs of $79,988 and $76,793, respectively. Conclusions: In line with ARROW trial results, this CE analysis showed that QW Kd70 is expected to provide considerable additional benefit in terms of LYs and QALYs gained compared with BIW Kd27. In the RRMM setting, QW Kd70 is cost-effective with ICERs below accepted willingness to pay thresholds in US and represents an efficient utilization of the health care budget.

scholarly journals Cost-effectiveness Analysis of Sequential Treatment of Abaloparatide Followed by Alendronate Versus Teriparatide Followed by Alendronate in Postmenopausal Women With Osteoporosis in the United States

2018 ◽  
Vol 53 (2) ◽  
pp. 134-143 ◽  
Author(s):  
Quang A. Le ◽  
Joel W. Hay ◽  
Russell Becker ◽  
Yamei Wang
Keyword(s):  
United States ◽  
Health Care ◽  
Cost Effectiveness ◽  
High Risk ◽  
The United States ◽  
Clinical Event ◽  
Risk Of Fracture ◽  
Life Years ◽  
The Us ◽  
Des Model

Background: The US Food and Drug Administration has recently approved abaloparatide (ABL) for treatment of women with postmenopausal osteoporosis (PMO) at high risk of fracture. With increasing health care spending and drug prices, it is important to quantify the value of newly available treatment options for PMO. Objective: To determine cost-effectiveness of ABL compared with teriparatide (TPTD) for treatment of women with PMO in the United States. Methods: A discrete-event simulation (DES) model was developed to assess cost-effectiveness of ABL from the US health care perspective. The model included three 18-month treatment strategies with either placebo (PBO), TPTD, or ABL, all followed by additional 5-year treatment with alendronate (ALN). High-risk patients were defined as women with PMO ⩾65 years old with a prior vertebral fracture. Baseline clinical event rates, risk reductions, and patient characteristics were based on the Abaloparatide Comparator Trial in Vertebral Endpoints (ACTIVE) trial. Results: Over a 10-year period, the DES model yielded average total discounted per-patient costs of $10 212, $46 783, and $26 837 and quality-adjusted life-years (QALYs) of 6.742, 6.781, and 6.792 for PBO/ALN, TPTD/ALN, and ABL/ALN, respectively. Compared with TPTD/ALN, ABL/ALN accrued higher QALYs at lower cost and produced an incremental cost-effectiveness ratio (ICER) of $333 266/QALY relative to PBO/ALN. In high-risk women, ABL/ALN also had more QALYs and less cost over TPTD/ALN and yielded an ICER of $188 891/QALY relative to PBO/ALN. Conclusion and Relevance: ABL is a dominant treatment strategy over TPTD. In women with PMO at high risk of fracture, ABL is an alternative cost-effective treatment.

scholarly journals The Clinical Impact and Cost-effectiveness of Measles-Mumps-Rubella Vaccination to Prevent Measles Importations Among International Travelers From the United States

2018 ◽  
Vol 69 (2) ◽  
pp. 306-315 ◽  
Author(s):  
Emily P Hyle ◽  
Naomi F Fields ◽  
Amy Parker Fiebelkorn ◽  
Allison Taylor Walker ◽  
Paul Gastañaduy ◽  
...  
Keyword(s):  
United States ◽  
Cost Effectiveness ◽  
Hot Spots ◽  
Cost Effective ◽  
The United States ◽  
Clinical Impact ◽  
Sensitivity Analyses ◽  
Best Value ◽  
Measles Outbreaks ◽  
The Impact

Abstract Background Measles importations and the subsequent spread from US travelers returning from abroad are responsible for most measles cases in the United States. Increasing measles-mumps-rubella (MMR) vaccination among departing US travelers could reduce the clinical impact and costs of measles in the United States. Methods We designed a decision tree to evaluate MMR vaccination at a pretravel health encounter (PHE), compared with no encounter. We derived input parameters from Global TravEpiNet data and literature. We quantified Riskexposure to measles while traveling and the average number of US-acquired cases and contacts due to a measles importation. In sensitivity analyses, we examined the impact of destination-specific Riskexposure, including hot spots with active measles outbreaks; the percentage of previously-unvaccinated travelers; and the percentage of travelers returning to US communities with heterogeneous MMR coverage. Results The no-encounter strategy projected 22 imported and 66 US-acquired measles cases, costing $14.8M per 10M travelers. The PHE strategy projected 15 imported and 35 US-acquired cases at $190.3M per 10M travelers. PHE was not cost effective for all international travelers (incremental cost-effectiveness ratio [ICER] $4.6M/measles case averted), but offered better value (ICER <$100 000/measles case averted) or was even cost saving for travelers to hot spots, especially if travelers were previously unvaccinated or returning to US communities with heterogeneous MMR coverage. Conclusions PHEs that improve MMR vaccination among US international travelers could reduce measles cases, but are costly. The best value is for travelers with a high likelihood of measles exposure, especially if the travelers are previously unvaccinated or will return to US communities with heterogeneous MMR coverage.

scholarly journals Cost-Effectiveness of Tafamidis Therapy for Transthyretin Amyloid Cardiomyopathy

Circulation ◽  
2020 ◽  
Vol 141 (15) ◽  
pp. 1214-1224 ◽  
Author(s):  
Dhruv S. Kazi ◽  
Brandon K. Bellows ◽  
Suzanne J. Baron ◽  
Changyu Shen ◽  
David J. Cohen ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Incremental Cost ◽  
Cost Effective ◽  
The United States ◽  
Quality Adjusted Life Year ◽  
Life Years ◽  
The Us ◽  
Amyloid Cardiomyopathy ◽  
Quality Adjusted Life

Background: In patients with transthyretin amyloid cardiomyopathy, tafamidis reduces all-cause mortality and cardiovascular hospitalizations and slows decline in quality of life compared with placebo. In May 2019, tafamidis received expedited approval from the US Food and Drug Administration as a breakthrough drug for a rare disease. However, at $225 000 per year, it is the most expensive cardiovascular drug ever launched in the United States, and its long-term cost-effectiveness and budget impact are uncertain. We therefore aimed to estimate the cost-effectiveness of tafamidis and its potential effect on US health care spending. Methods: We developed a Markov model of patients with wild-type or variant transthyretin amyloid cardiomyopathy and heart failure (mean age, 74.5 years) using inputs from the ATTR-ACT trial (Transthyretin Amyloidosis Cardiomyopathy Clinical Trial), published literature, US Food and Drug Administration review documents, healthcare claims, and national survey data. We compared no disease–specific treatment (“usual care”) with tafamidis therapy. The model reproduced 30-month survival, quality of life, and cardiovascular hospitalization rates observed in ATTR-ACT; future projections used a parametric survival model in the control arm, with constant hazards reduction in the tafamidis arm. We discounted future costs and quality-adjusted life-years by 3% annually and examined key parameter uncertainty using deterministic and probabilistic sensitivity analyses. The main outcomes were lifetime incremental cost-effectiveness ratio and annual budget impact, assessed from the US healthcare sector perspective. This study was independent of the ATTR-ACT trial sponsor. Results: Compared with usual care, tafamidis was projected to add 1.29 (95% uncertainty interval, 0.47–1.75) quality-adjusted life-years at an incremental cost of $1 135 000 (872 000–1 377 000), resulting in an incremental cost-effectiveness ratio of $880 000 (697 000–1 564 000) per quality-adjusted life-year gained. Assuming a threshold of $100 000 per quality-adjusted life-year gained and current drug price, tafamidis was cost-effective in 0% of 10 000 probabilistic simulations. A 92.6% price reduction from $225 000 to $16 563 would be necessary to make tafamidis cost-effective at $100 000/quality-adjusted life-year. Results were sensitive to assumptions related to long-term effectiveness of tafamidis. Treating all eligible patients with transthyretin amyloid cardiomyopathy in the United States with tafamidis (n=120 000) was estimated to increase annual healthcare spending by $32.3 billion. Conclusions: Treatment with tafamidis is projected to produce substantial clinical benefit but would greatly exceed conventional cost-effectiveness thresholds at the current US list price. On the basis of recent US experience with high-cost cardiovascular medications, access to and uptake of this effective therapy may be limited unless there is a large reduction in drug costs.

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